BURLINGTON, Mass., Sept. 10, 2014 /PRNewswire/ -- Decision Resources Group finds that the first three emerging disease-modifying therapies (DMTs)—PTC Therapeutics' Translarna (ataluren), Prosensa's drisapersen and Sarepta's eteplirsen—will likely receive regulatory approval for Duchenne muscular dystrophy (DMD) in the EU5 markets (France, Germany, Italy, Spain and the United Kingdom) and/or the United States during the 2013-2023 study period. Translarna was conditionally approval by the European Medicines Agency in August 2014 and is the only therapy intended to correct dystrophin nonsense mutations in DMD. In contrast, drisapersen and eteplirsen will directly compete for DMD patients with mutations addressable by skipping translation of dystrophin exon 51. Together, eteplirsen, drisapersen and ataluren will address mutations present in less than one third of DMD patients. Further, experts are concerned that even within the eligible subpopulations the individual responses may vary considerably. They are therefore eager for additional treatment options to maintain and restore muscle fibers by replenishing Dystrophin levels and/or compensate for its absence by increasing overall muscle regeneration.

Other key findings from the Niche Markets and Rare Diseases: Muscular Dystrophy report:

  • Becker muscular dystrophy (BMD) and Limb-Girdle muscular dystrophy (LGMD): Although both BMD and DMD are caused by mutations in the dystrophin gene, BMD is a milder form of dystrophy than DMD. The LGMDs are a group of MDs mostly affecting hips and shoulders. Interviewed experts point out that there are no DMTs available for BMD or LGMDs and other than treatment of the associated cardiomyopathy with ACE inhibitors, management of these MDs is primarily through best supportive care.
  • The six-minute walk test (6MWT): Physicians agree that performing the 6MWT consistently is very challenging in young boys with DMD. Despite these difficulties, experts agree that a primary end point evaluating functional motor skills is crucial for assessing treatment efficacy. Lacking a better surrogate, thought leaders believe that the 6MWT will be the basis for regulatory approval and physician prescribing of early-stage DMD agents in the foreseeable future.

Comments from Decision Resources Group Analyst Seamus Levine-Wilkinson, Ph.D.:

  • "Interviewed experts have closely followed the development of "exon skipping" therapies and are eager to begin prescribing them to their DMD patients. Moreover, thought leaders hope that successful commercialization of drisapersen and/or eteplirsen will pave the way for additional dystrophin exon skipping agents."
  • "Glucocorticoids will continue to be an important therapy for DMD through 2023, while the three new DMTs will target only a portion of DMD patients and will likely be used in addition to the glucocorticoids. However, Santhera's idebenone is expected to launch in the United States and Europe as a treatment option for DMD patients who are not eligible for glucocorticoid therapy."

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